CSL Behring Gains Approval to License Hizentra® in Japan for the Treatment of Primary Immunodeficiency and Secondary Immunodeficiency

CSL Behring announced today that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Hizentra® (Immune Globulin Subcutaneous [Human]) for the treatment of primary immunodeficiency (PID) and for the treatment of secondary immunodeficiency (SID). Hizentra is now the first and only 20 percent subcutaneous immunoglobulin (SCIg) therapy in the world for the treatment of these conditions, both of which belong to group of rare and serious diseases of the immune system. The Japanese MHLW approval marks the first time any SCIg therapy has been approved for use in Japan.

27 Sep 2013

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CSL Behring announced today that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Hizentra® (Immune Globulin Subcutaneous [Human]) for the treatment of primary immunodeficiency (PID) and for the treatment of secondary immunodeficiency (SID). Hizentra is now the first and only 20 percent subcutaneous immunoglobulin (SCIg) therapy in the world for the treatment of these conditions, both of which belong to group of rare and serious diseases of the immune system. The Japanese MHLW approval marks the first time any SCIg therapy has been approved for use in Japan.

The approval was based on a Phase III study of 25 Japanese patients that found when patients converted from intravenous immunoglobulin (IVIg) treatment to dose-equivalent Hizentra therapy, serum IgG (immunoglobulin), trough concentrations increased to levels higher than those seen with their previous IVIG therapy. Results showed that Hizentra provided effective passive immunity in adults and children, which controlled most recurrent infections and improved patients’ overall quality of life.

“We are extremely pleased to have secured this important regulatory approval for Hizentra in Japan,” said Christopher Church, Vice President and General Manager, CSL Behring Asia Pacific Ltd. “For some years, Hizentra has been available in several areas of the world, making a positive difference for patients and healthcare professionals who seek innovative options for treating PID and SID. Today, it is gratifying to bring such an option to Japan, where Hizentra can address a clear, unmet medical need.”

Study Design and Key Findings
In a prospective multicenter, open-label, single-arm Phase III study of Hizentra, 25 Japanese patients with PID, who required IgG replacement therapy, were evaluated following an IVIg treatment period. Patients received three infusions of IVIG therapy, followed by a 12-week wash-in/wash-out period where treatment was converted to Hizentra and a 12-week efficacy period. The primary endpoint was achievement of the total serum IgG trough levels with SCIg therapy as compared to the preceding IVIg treatment period. Secondary efficacy and safety endpoints included all infections and local reactions to treatment and adverse events (AEs).

Findings demonstrated that Hizentra produced serum IgG trough concentrations higher than those seen during previous IVIg therapy. Mean serum IgG levels increased from 6.51 (1.32) g/L in the IVIG period to 7.28 (1.47) g/L in the SCIg efficacy period. During the efficacy period, no serious bacterial infections were reported; 11 patients experienced a non-serious infection. Ninety-six percent of the patients enrolled experienced at least one AE; most common AEs were local reactions of mild intensity. No related serious AEs were reported.

About Primary and Secondary
Immunodeficiencies constitute a group of more than 150 diseases that affect the cells, tissues and proteins of the immune system. In people with PID or SID, the immune system is either absent or functioning inadequately, leaving them more susceptible to infection. In children, especially, infections may not improve with treatment as expected and may keep returning. As a result, patients may face repeated rounds of antibiotics and hospitalization for treatment. Repeated infections can lead to organ damage, which, over time, can become life threatening. Collectively, immunodeficiencies affect an estimated 10 million people worldwide; the incidence is estimated to be 1 in 10,000.

For patients with PID or SID, immunoglobulin replacement therapy with a product such as Hizentra can help treat existing or chronic infections and prevent new infections from occurring. No single treatment works for every type of PID or SID, but infusions of replacement antibodies (immunoglobulins) can help supplement the immune system to prevent infection in nearly 75 percent of PID cases that are due to antibody deficiencies. Immunoglobulin is a blood component that has become standard immune replacement therapy for people living with PID, and nearly 70 percent of PID patients receive Ig replacement therapy. Since the 1980s, the first-line therapy for most PID or SID patients has been intravenous immunoglobulin (IVIg), which immunoglobulin is delivered through a needle into the vein. Some patients, however, cannot easily tolerate intravenous infusions due to serious side effects or poor venous access. Hizentra allows patients to use a small, portable pump to self-administer their infusions by injection under the skin (subcutaneous administrations). For more information on PID, please visit www.cslbehring.com.

About HizentraHizentra (Immune Globulin Subcutaneous [Human]), the first and only 20 percent SCIg developed for subcutaneous use, is already approved in North America and Europe. It is stable at 25 degrees Celcius for at least 30 months due to formulation with L-proline. In the United States, Hizentra is indicated for the treatment of patients with PID, and contraindicated in individuals with a history of anaphylactic or severe systemic response to immune globulin preparations or components of Hizentra, and in persons with selective immunoglobulin A deficiency who have known antibody against IgA and a history of hypersensitivity. For more information, including full U.S. prescribing information, visit http://www.hizentra.com/. In 2011, the European Commission granted marketing authorization for Hizentra for treating patients diagnosed with PID as well as secondary immunodeficiencies. The authorization is valid for all 29 European/European Economic Area member states.

About CSL Behring
CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients' lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company's therapies are used in the treatment of immune deficiency disorders, hereditary angioedema, hemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world's largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit www.cslbehring.com